Sonidegib, a hedgehog pathway inhibitor, has been approved for treating patients with locally advanced basal cell carcinoma (BCC) that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy, the Food and Drug Administration announced on July 24. The drug is taken once a day, at a recommended dose of 200 mg on an empty stomach and will be marketed as Odomzo by Novartis Pharmaceuticals. Approval was based on a study that found the objective response rate to be 58% among patients treated with the 200-mg dose; an effect that lasted for about 2-19 months, according to the FDA.
Dr. Richard Pazdur
“Our increasing understanding of molecular pathways involved in cancer has led to approvals of many oncology drugs in difficult-to-treat diseases for which few therapeutic options previously existed,” Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in the statement. “Thanks to a better understanding of the hedgehog pathway, the FDA has now approved two drugs for the treatment of basal cell carcinoma just in the last 3 years,” he added.
The first drug to treat locally advanced and metastatic basal cell carcinoma was vismodegib, marketed as Erivedge by Genentech in 2012.
The most common adverse events associated with the 200-mg dose include muscle spasms, alopecia, dysgeusia, fatigue, nausea, musculoskeletal pain, diarrhea, decreased weight, decreased appetite, myalgia, abdominal pain, headache, pain, vomiting, and pruritus (itching). It has also been associated “with rare reports” of rhabdomyolysis, muscle spasms, and myalgia, according to the FDA.
The prescribing information includes a boxed warning about the risk of embryo-fetal toxicity, and includes recommendations to verify that women of reproductive potential are not pregnant before starting treatment and to use effective contraception during treatment and for at least 20 months after the last dose. Men on this treatment should also be advised about “the potential risk of exposure through semen and to use condoms with a pregnant partner or a female partner of reproductive potential during treatment,” and for at least 8 months after stopping treatment.
More information on the approval is available on the FDA website at www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm455865.htm.
Exposed pregnancies should be reported to Novartis at 888-669-6682. Serious adverse events associated with sonidegib should be reported to the FDA’s MedWatch program at 800-332-1088 or www.fda.gov/Safety/MedWatch.